Gene therapy treatments for rare diseases are being developed, but getting them out of the lab has proved challenging.

What began as a desperate plea of hope has become a story of healing, progress and overwhelming gratitude for a Wake County ...

Severe Combined Immunodeficiency (SCID) is a rare, life-threatening immunological disorder characterized due to the lack of B-lymphocyte and T-lymphocyte functioning. Sporadic (occasional) ...

Building on its contributions to newborn screening globally, PerkinElmer’s latest FDA authorized assay enables the simultaneous detection of SMA and SCID SMA is a leading genetic cause of infant death ...

Overall and event-free survival of 100 and 95%, respectively, seen with autologous CD34+ hematopoietic stem-cell lentiviral gene therapy. HealthDay News — Autologous CD34+ hematopoietic stem-cell ...

David, born with immune deficiency syndrome, plays in the enclosed plastic environment in which he must live to survive. Doctors at the Texas Children's Hospital search for a way to stimulate his ...

St. Jude researchers successfully used a deactivated HIV virus to successfully treat 'bubble boy' disease in a groundbreaking study. Pictured above is one of the 12 participants. (Photo: St. Jude) ...

UCLA researchers are restarting a study to give a gene therapy to kids with an ultra-rare disease. While the one-time therapy is safe and effective, the drug industry has been uninterested. The ...

An investigational gene therapy showed sustained clinical efficacy for children born with ADA-SCID. In a study of 62 children, overall survival after treatment was 100% and event-free survival was 95% ...

HOUSTON, TEXAS - DECEMBER 23: A sign at the entrance to William P. Hobby Airport on December 23, 2024 in Houston, Texas. (Photo by Kirby Lee/Getty Images) Read full article: Plane crashes in Tennessee ...

Read full article: 🍬Sweets, suites combine new characters with nostalgic Disney World themes on Treasure cruise ship Orange County Comptroller Phil Diamond; Elections Supervisor Glen Gilzean. ST.